Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Familial Hypercholesterolemia - Heterozygous

In brief

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).

Key facts

Study ID

NCT06597019

Run by

Novartis Pharmaceuticals

People needed

60

Starts

2024-12-09

Expected to finish

2029-04-15

Last updated by the study team

2026-05-15

Who can join

Age: 6 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female participants, 6 to <12 years of age at screening
• HeFH diagnosed either by genetic testing or on phenotypic criteria
• Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
• For participants 8 to <12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants <8 years, the use of background lipid-lowering treatment is based on investigator's discretion.
• Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation.

You may not qualify if…

• Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
• Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
• Homozygous familial hypercholesterolemia (HoFH)
• Body weight <16 kg at the screening and/or randomization (Day 1) visit
• Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation >3x ULN, or total bilirubin elevation >2x ULN (except patients with Gilbert's syndrome)
• Pregnant or nursing females
• Recent and/or planned use of other investigational medicinal products or devices

Where it is running

• UC San Francisco Medical Center — San Francisco, California, United States (enrolling)
• Novartis Investigative Site — Marseille, France (enrolling)
• Childrens National Hospital — Washington D.C., District of Columbia, United States (enrolling)
• Excel Medical Clinical Trials LLC — Boca Raton, Florida, United States (enrolling)
• Icahn School of Med at Mt Sinai — New York, New York, United States (enrolling)
• UC San Francisco Medical Center — San Francisco, California, United States (enrolling)
• Primary Childrens Medical Center — Salt Lake City, Utah, United States (enrolling)
• Virginia Commonwealth University — Richmond, Virginia, United States (enrolling)
• West Virginia Childrens Hospital — Morgantown, West Virginia, United States (enrolling)
• West Virginia Childrens Hospital — Morgantown, West Virginia, United States (enrolling)
• Novartis Investigative Site — Buenos Aires, Argentina (enrolling)
• Novartis Investigative Site — CABA, Argentina (enrolling)
• Novartis Investigative Site — Salzburg, Austria (enrolling)
• Novartis Investigative Site — Vienna, Austria (enrolling)
• Novartis Investigative Site — Brussels, Belgium (enrolling)
• Novartis Investigative Site — Leuven, Belgium (enrolling)
• Novartis Investigative Site — Fortaleza, Ceará, Brazil (enrolling)
• Novartis Investigative Site — Rio de Janeiro, Rio de Janeiro, Brazil (enrolling)
• Novartis Investigative Site — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)
• Novartis Investigative Site — São Paulo, São Paulo, Brazil (enrolling)
• Novartis Investigative Site — Beijing, Beijing Municipality, China (enrolling)
• Novartis Investigative Site — Shanghai, China (enrolling)
• Novartis Investigative Site — Prague, Czechia (enrolling)
• Novartis Investigative Site — Prague, Czechia (enrolling)
• Novartis Investigative Site — Nantes, France (enrolling)

Full record on ClinicalTrials.gov

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